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It is in a class of medicines called erythropoiesis-stimulating agents (ESAs).
Darbepoetin alfa
Darbepoetin alfa:
It is in a class of medicines called erythropoiesis-stimulating agents (ESAs). Darbepoetin alfa can help improve red blood cell counts in bone marrow failure patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in…
Drug last updated
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Cyclosporine
Cyclosporine:
Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure.
is used along with
antithymocyte globulin
antithymocyte globulin:
ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the…
Drug last updated
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It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely.
Azacitidine
Azacitidine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts…
Drug last updated
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Alemtuzumab
Alemtuzumab:
Alemtuzumab is a drug used in the treatment of chronic lymphocytic leukemia (CLL), cutaneous T-cell lymphoma (CTCL) and T-cell lymphoma under the trade names Campath, MabCampath and Campath-1H, and in the treatment of multiple sclerosis as Lemtrada. It is also used in some conditioning regimens for…
is a drug used in the treatment of chronic lymphocytic…
Drug last updated
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Cyclophosphamide
Cyclophosphamide:
Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system.
is in a class of medications called alkylating agents. When used to…
Drug last updated
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Romiplostim
Romiplostim:
Romiplostim is in a class of medications called thrombopoietin receptor agonists. It works by causing the cells in the bone marrow to produce more platelets. Romiplostim injection is used to increase the number of platelets enough to lower the risk of bleeding, but it is not used to increase the…
is in a class of medications called thrombopoietin receptor…
Drug last updated
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Learn about
biosimilars
biosimilars:
What is a Biosimilar?
A biosimilar is a type of biologic medication. The term “biosimilar” means that the medication is highly similar to the already approved brand name biologic (“originator” medication). Biosimilars are not generic versions of the original biologic.
Difference Between…
, biosimilar medications, and their use for
bone marrow…
Webinar last updated
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Iptacopan (LNP023)
Iptacopan (LNP023):
Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway. It is currently in clinical development for treating PNH.
is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway. It is currently…
Drug last updated
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Cemdisiran (ALN-CC5)
Cemdisiran (ALN-CC5):
Cemdisiran (ALN-CC5) is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for the treatment of complement-mediated diseases.
is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for…
Drug last updated
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Pozelimab
Pozelimab:
Pozelimab is a monoclonal antibody inhibiting the pathway associated with haemolysis of red blood cells. The drug is intended to decrease LDH levels and the incidence of breakthrough haemolysis.
is a
monoclonal antibody
monoclonal antibody:
A type of protein called an antibody that is engineered to look for a specific substance in the body. There are many…
Drug last updated
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Danicopan (ACH-4471)
Danicopan (ACH-4471):
Danicopan (ACH-4471) inhibits alternative pathway of complement (APC) activity, and has potential to block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria (PNH).
inhibits alternative pathway of complement (APC) activity, and has potential to block the alternative pathway of complement in
paroxysmal nocturnal…
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APG-2575 is a novel, orally administered small-molecule Bcl-2‒selective inhibitor. APG-2575 is designed to treat hematologic malignancies and solid tumors by selectively blocking antiapoptotic protein Bcl-2 to restore the normal
apoptosis
apoptosis:
Programmed cell death.
process in cancer cells.
Drug last updated
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Alrizomadlin is an orally administered, selective, small-molecule inhibitor of the MDM2 protein. Alrizomadlin has strong binding affinity to MDM2 and is designed to activate tumor suppression activity of p53 by blocking the MDM2-p53 protein-protein interaction.
Drug last updated
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Cytarabine (ara-C)
Cytarabine (ara-C):
Cytarabine (ara-C) is a chemotherapy drug. This medicine reduces the growth of cancer cells and can suppress the immune system. It is used for treating leukemias or lymphomas. It is often given with other cancer drugs.
is a
chemotherapy
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People…
Drug last updated
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A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to IMMUNOPHILINS.
Sirolimus
Sirolimus:
A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of…
Drug last updated
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Ravulizumab-cwvz
Ravulizumab-cwvz:
Ravulizumab-cwvz (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade. ULTOMIRIS® is a prescription medicine called a…
(Ultomiris®) is a drug approved by the U.S. Food…
Drug last updated
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Prednisone
Prednisone:
Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own.
How does it work?
For PNH, it may be used to decrease…
a man-made version of a
hormone
hormone:
A part of the endocrine…
Drug last updated
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Daunorubicin
Daunorubicin:
Daunorubicin is in a class of medications called anthracyclines. It works by slowing or stopping the growth of cancer cells in your body. It is used in combination with other chemotherapy drugs to treat a certain type of acute myeloid leukemia (AML).
is in a class of medications called anthracyclines. It works by slowing or stopping the growth…
Drug last updated
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